rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study

NCT03829449 | Terminated Phase 3 Results FDA Drug

• 1 other identifier: AK581
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

Long term management of patients with complement related diseases including Paroxysmal Nocturnal Haemoglobinuria and Atypical Haemolytic Uraemic Syndrome

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Outcome Measures

Primary Outcomes (1)

• Long Term Safety and Efficacy of rVA576 (Coversin) Therapy Assessed by AEs, SAEs, Standard Lab Tests and ECG Results.

  To determine the safety profile of long-term rVA576 (Coversin) treatment as assessed by AEs, SAEs, Standard Lab tests and ECG results.

  On entry and every 3 months thereafter, for the duration of the study (approximately 3 years and 5 months)

Secondary Outcomes (12)

• Proportion of Subjects With Thrombotic and Haemolytic Event Free Status During Each 3month Time Period Since the Start of the Study.

  On entry and every 3 months thereafter, for the duration of the study (approximately 3 years and 5 months)

• Time to Thrombotic or Haemolytic Event Since Joining This Study.

  Approximately 3 years and 5 months

• Proportion of Subjects Who Require PRBC Transfusion During Each 3-month Period Since the Start of the Study and Over the Entire Period of the Study

  On entry and every 3 months thereafter, for the duration of the study (approximately 3 years and 5 months)

• Time to First Transfusion Since Joining the Study.

  approximately 3 years and 5 months

• Proportion of Subjects With no Adverse Change in Overall Scores of Quality of Life Using the EORTC QLQ-C30, the EQ-5D-5L and FACIT-F Instruments at Each 3-month Time Period Since the Start of the Study.

  Every 3 months up to 39 months

Study Arms (1)

rVA576 Coversin

EXPERIMENTAL

The study population will consist of patients who have completed participation in clinical trials under other Akari protocols and who wish to continue to receive rVA576 (Coversin) for up to 4 years.

Drug: rVA576

Interventions

rVA576 DRUG

The study population will consist of patients who have completed participation in clinical trials under other Akari protocols and who wish to continue to receive rVA576 (Coversin).

Also known as: nomacopan

rVA576 Coversin

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients 18 years and above treated with rVA576 (Coversin) under other Akari clinical trial protocols and wish to remain on rVA576 (Coversin) at the conclusion of that trial.
• In the opinion of the treating responsible clinician patient is receiving clinical benefit from continued treatment with study drug.
• Evidence of sustained complement inhibition by CH50 assay. .
• Women of childbearing potential (WOCBP) must agree to use effective contraception consistently throughout the study and have a negative pregnancy test at screening and a negative urine pregnancy test per the schedule of visits. Women cannot donate their eggs. Women are considered post-menopausal and not of childbearing potential if they have had 12 months of amenorrhea or have had surgical bilateral oophorectomy (with or without hysterectomy) or tubal ligation at least six weeks previously.
• Males with a childbearing potential partner must agree to use effective contraception consistently OR have had a vasectomy
• Weight ≥50-100kg
• Willing to receive appropriate prophylaxis against Neisseria meningitidis infection, by both immunisation and continuous or intermittent antibiotics
• The patient is willing to give voluntary written informed consent
• The patient is willing in the process of preparation and self-administration of the study drug.

You may not qualify if:

• Patient experienced any safety event in the previous study protocol, which puts the patient at unacceptable risk in current protocol as judged by the investigator and sponsor.
• Patient is unwilling to complete the Quality of Life instruments and diary card
• Active meningococcal infection (section 4.3.1 for additional information)
• Any other reason for which, in the opinion of the Investigator, it would not be in the interests of the patient to remain on rVA576 (Coversin).
• If female, the subject is pregnant or lactating or intending to become pregnant before, during, or within 90 days after last dose; or intending to donate ova during such time period.
• If male, the subject intends to donate sperm while on the study this study or for 90 days after last dose.
• Failure to satisfy the Investigator of fitness to participate for any other reason or any other condition which, in the opinion of the investigator, could increase the subject's risk by participating in the study or confound the outcome of the study.
• Use of prohibited medication
• The subject has a history of drug abuse (defined as any illicit drug use) or a history of alcohol abuse.
• Participation in other clinical trials with investigational product.

Sponsors & Collaborators

• AKARI Therapeutics: lead

Study Sites (1)

Instytut Hematologii i Transfuzjologii

Warsaw, 02-776, Poland

Location

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Limitations and Caveats

Due to the early termination of this study, only a subset of the outputs planned were produced. Parent trial locked databases were not pooled with the AK581 database, limiting the scope of the analyses that could be produced. The 'parent trial baseline' could not be used in calculations.

Results Point of Contact

• Title: Dr Wynne Weston-Davies
• Organization: Akari Therapeutics Plc

wynne.weston-davies@akaritx.com

0208 004 6106

Study Officials

• Wynne Weston Davies

  AKARI Therapeutics

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Open-label, non-comparative

Study Record Dates

• First Submitted: December 20, 2018
• First Posted: February 4, 2019
• Study Start: March 13, 2017
• Primary Completion: August 29, 2020
• Study Completion: August 29, 2020
• Last Updated: April 10, 2025
• Results First Posted: March 12, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

PL (1)